Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors

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What is this study about?

This clinical trial focuses on studying different treatment approaches for patients with Hemophilia A, a condition where blood does not clot properly due to a lack of a specific protein called factor VIII. The study involves several treatments, including Hemlibra (also known as emicizumab), Nuwiq (simoctocog alfa), FEIBA (factor VIII inhibitor bypassing fraction), Wilate (human coagulation factor VIII and human von Willebrand factor), OCTANATE (human coagulation factor VIII), and NovoSeven (eptacog alfa, activated). These treatments are administered either through injections or infusions.

The purpose of the study is to evaluate the effectiveness of these treatments in patients with Hemophilia A who have developed inhibitors, which are antibodies that reduce the effectiveness of factor VIII treatments. The study is divided into three groups. Groups 1 and 2 will focus on evaluating the success of immune tolerance induction (ITI), a process aimed at reducing inhibitor levels, while Group 3 will assess the annualized bleeding rate (ABR) compared to the other groups. Participants will receive their assigned treatments over a period of up to 60 weeks, with regular monitoring to assess their response to the treatment.

Throughout the study, participants will be monitored for the frequency and severity of bleeding episodes, the number of infusions needed to control bleeding, and any side effects or adverse reactions. The study aims to provide valuable insights into the best treatment strategies for managing Hemophilia A in patients with inhibitors, ultimately improving their quality of life and treatment outcomes.

1 joining the study

Upon joining the study, participants are assigned to one of three groups based on their treatment needs and history with hemophilia A.

Participants must have a historical inhibitor titre of at least 0.6 Bethesda units per milliliter (BU/mL).

2 initial treatment phase

Participants in Groups 1 and 2 undergo immune tolerance induction (ITI) therapy using products such as Nuwiq, Octanate, or Wilate.

During ITI, some participants may also receive prophylactic therapy with emicizumab, activated prothrombin complex concentrate (aPCC), or recombinant activated factor VII (rFVIIa).

3 medication administration

Emicizumab is administered subcutaneously, with available dosages including 30 mg/mL and 150 mg/mL solutions for injection.

Nuwiq, Octanate, and Wilate are administered intravenously, with various dosages available, such as 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, and 3000 IU.

4 monitoring and evaluation

Participants are regularly monitored to evaluate the success of ITI, which includes achieving an inhibitor titre of less than 0.6 BU/mL for at least two consecutive measurements.

The recovery and half-life of factor VIII are also assessed to determine the effectiveness of the treatment.

5 bleeding rate assessment

For Group 3, the annualized bleeding rate (ABR) is evaluated and compared with the ABR in Groups 1 and 2.

The frequency and severity of bleeding episodes, including joint bleeds, are recorded over time.

6 follow-up and outcomes

Participants who achieve complete ITI success are followed up to monitor for any relapses of factor VIII inhibitors.

The study also tracks adverse drug reactions (ADRs), thrombotic events, and treatment costs.

Who Can Join the Study?

Participants can be of any age at the time of joining the trial.
Must be male with Hemophilia A, which is a condition where blood doesn’t clot properly, of any severity.
Must have a past inhibitor titre of at least 0.6 Bethesda units per milliliter (BU/mL). An inhibitor titre is a measure of substances in the blood that can interfere with treatment.
Can include those who have not succeeded in previous ITI attempts. ITI stands for Immune Tolerance Induction, a treatment to help the body accept clotting factor treatments.
Must be undergoing ITI with specific treatments like Nuwiq®, octanate®, or wilate®.
Can also be receiving preventive therapy with emicizumab, aPCC, or rFVIIa. These are medications used to help prevent bleeding episodes.
Participants or their parent(s)/legal guardian(s) must be able to give signed informed consent, meaning they understand and agree to the trial’s terms.

Who Cannot Join the Study?

Only male participants are allowed, so females cannot participate.
Participants must have Haemophilia A, a condition where blood doesn’t clot properly.
Participants must be within certain age ranges, which are not specified here.
Participants must not belong to a vulnerable population, which means they should not be in a group that needs special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Oslo Universitetssykehus HF	Oslo	Norway

Other Sites

Site Name	City	Country
Hospital Universitario San Juan De Alicante	Sant Joan D'alacant	Spain
Region Skane Skanes Universitetssjukhus	Lund	Sweden
KBC Zagreb	Zagreb	Croatia
Virgen del Rocío University Hospital	Sevilla	Spain
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Justus-Liebig-Universitaet Giessen	Giessen	Germany
HZRM Haemophilie-Zentrum Rhein Main GmbH	Mörfelden-Walldorf	Germany
Specialized Hospital For Active Treatment Of Hematological Diseases EAD	Sofia	Bulgaria
Gxicdbvkflykxmmtu Rdmwoiiuyu Axolxppjfnturbbvuzt Dqu moov Hrwacrdfp Rofv Fgkvjrwibms fpev Turwcacwkiwrzddbkpk Hqsftquxvkfxsfcl Dty moyy Sjovr Haoeolq Fdvobswcvos fkpl Tqcmfezfajrmupkcypc Hfmtyyvvlyjeekpg Dvp mrjg Gfkxsrzk Kymgpda Feiczmxp fhtp Ltiamnheyjgexdszkvik Hasuafjdplzyarbf	Duisburg	Germany
Hphzznkp Uitygagdpp Caxodrr Hayeadvw	Helsinki	Finland
Uhmlukaqualzfmkyoauad Mtpvzklh Aed	Munster	Germany
Hqsjzzre Vuqz dftxzqsl	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Bulgaria	Not recruiting	17.03.2020
Croatia	Recruiting	17.03.2020
Finland	Not recruiting	17.03.2020
Germany	Recruiting	17.03.2020
Norway	Recruiting	17.03.2020
Spain	Recruiting	17.03.2020
Sweden	Recruiting	17.03.2020

Trial locations

Investigated drugs:

Emicizumab is a medication used in the trial to help prevent bleeding episodes in patients with Hemophilia A. It works by mimicking the activity of a protein called factor VIII, which is missing or not working properly in these patients. This medication helps the blood to clot more effectively, reducing the risk of bleeding.

Factor VIII Concentrate is used in the trial to replace the missing or deficient factor VIII in patients with Hemophilia A. This therapy helps to control and prevent bleeding episodes by providing the necessary clotting factor that the body lacks. It is an essential part of the treatment for managing Hemophilia A.

ITI Therapy (Immune Tolerance Induction) is a treatment approach used in the trial for patients who have developed inhibitors against factor VIII. The goal of ITI therapy is to train the immune system to accept factor VIII without reacting against it, allowing patients to use factor VIII concentrates effectively to manage their condition.

Haemophilia A – Haemophilia A is a genetic disorder characterized by a deficiency in clotting factor VIII, which leads to prolonged bleeding. This condition primarily affects males and is inherited in an X-linked recessive pattern. Individuals with Haemophilia A may experience spontaneous bleeding episodes, particularly into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to chronic joint damage and reduced mobility. The severity of the disease varies, with some individuals experiencing frequent bleeding episodes and others having milder symptoms. Regular monitoring and management are essential to prevent complications associated with bleeding.

Trial ID:

2024-516741-39-00

Protocol code:

MOTIVATE

NCT ID:

NCT04023019

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors

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